NCT03914209

Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Patients With Hemophilia

Status unknown Last updated 2 September 2021

What this trial tests

trial testing EHL clotting factor in Haemophilia in 40 participants. Status unknown.

Timeline

2 June 2020

Primary endpoint
21 September 2021

15 October 2021

Quick facts

Lead sponsor	Investigación en Hemofilia y Fisioterapia
Status	Status unknown
Study type	OBSERVATIONAL
Enrollment	40
Start date	2 June 2020
Primary completion	21 September 2021
Estimated completion	15 October 2021
Sites	1 location across Spain

Drugs / interventions tested

• EHL clotting factor

Conditions studied

• Haemophilia — all drugs for Haemophilia →

Sponsor

Investigación en Hemofilia y Fisioterapia

Who can join

Adults 18 to 65, male only, with Haemophilia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Background. Hemophiliac arthropathy is the main long-term physical damage in patients with hemophilia, causing disability and functional limitations.

Objective. Assessment of the efficacy of a prophylactic treatment with EHL products for improving the musculoskeletal health of adult patients with hemophilia.

Study Design. Multicenter observational clinical study. Method. 40 patients with hemophilia A will be included in this study. Patients will be recruited from 5 centers located in various regions of Spain (Community of Madrid, Galicia, Community of Valenciana, Málaga and Vizcaya). The dependent variables will be: bleeding frequency (self-report of bleeding), changes in the dosage of factor VIII with EHL products (rFVIIIFc), pain (measured with the visual analog scale and a pressure Algometer), degree of kinesiophobia (Tampa Scale of Kinesiophobia), degree of adherence to treatment (Veritas-Pro scale), joint health (using the Hemophilia Joint Health Score), muscle strength (using a dynamometer) and functionality (using the 6-Minute Walking Test). Three evaluations will be carried out: baseline, at 6 months, and at the end of the study period, at 12 months. The evolution of quantitative variables shall be analyzed by parametric tests (t-student test) or non-parametric tests (Wilcoxon test). Pearson's correlation coefficient shall be used to obtain the correlation between the dependent and independent variables. By means of a linear regression analysis we will record the percentage influence of the clinical variables relative to the dependent variables measured in this study Expected results. The aim is to establish the effectiveness of the prophylactic treatment in the improvement of the state health, joint pain, muscle strength and functionality in patients with hemophilia.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Recombinant factor VIII Fc for the treatment of haemophilia A.
   Hermans C, Mancuso ME, Nolan B, Pasi KJ. · · 2021 · cited 16× · PMID 33650192 · DOI 10.1111/ejh.13610
2. LongHest project: A prospective, observational study of extended half-life treatment in the musculoskeletal health of patients with severe haemophilia A.
   Cuesta-Barriuso R, Pérez-Llanes R, Donoso-Úbeda E, Ucero-Lozano R. · · 2022 · cited 5× · PMID 35561281 · DOI 10.1111/hae.14592
3. Changes in Quality of Life, Adherence, and Kinesiophobia in Patients with Hemophilia Treated with Extended Half-Life Treatment: Final Results of the LongHest Project.
   Ucero-Lozano R, Pérez-Llanes R, Cuesta-Barriuso R, Donoso-Úbeda E. · · 2024 · cited 2× · PMID 39065686 · DOI 10.3390/ph17070835